A close relative of the microorganism that causes tuberculosis in humans has been found to form spores. This is a sensational finding because researchers have long been convinced that these kinds of bacteria–the mycobacteria–were incapable of forming spores. Leif Kirsebom’s research group at Uppsala University now has photographic proof, obtained...
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Houston, Texas – In a report published in Nature Cell Biology, researchers at Baylor College of Medicine, Texas Children’s Hospital, the University of Manitoba and collaborating institutions revealed an unexpected way in which the protein OTX2 drives the progression of medulloblastoma – the most common aggressive childhood brain cancer. The...
MINNEAPOLIS – OX2 Therapeutics, Inc., a privately held Minneapolis based company, announced today encouraging early results from phase 1 human trials of their new cancer treatment. Developed to combat recurrent high-grade brain tumors, the treatment utilizes a newly developed peptide inhibitor. “OX2 works to explore the therapeutic potential of the new immune...
LEXINGTON, Mass. and AMSTERDAM, Netherlands— uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the company reached agreement with the U.S. Food and Drug Administration (FDA) on key elements of an Accelerated Approval pathway for AMT-130. “We are...
LEXINGTON, Mass. and AMSTERDAM — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington’s disease, a rare, inherited neurodegenerative disorder...
LEXINGTON, Mass. and AMSTERDAM — uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced updated interim data including up to 30 months of follow-up from 39 patients enrolled in the ongoing U.S. and European Phase I/II clinical trials of AMT-130 for...
LEXINGTON, Mass. and AMSTERDAM, Netherlands — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the completion of enrollment in the first cohort of the Phase I/IIa trial of AMT-191, an investigational gene therapy for the treatment of Fabry disease....
LEXINGTON, Mass. and AMSTERDAM, the Netherlands — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced positive topline data from the pivotal Phase I/II study of AMT-130 for the treatment of Huntington’s disease. The study met its prespecified primary endpoint,...
Nijmegen, The Netherlands – TB is a major threat to public health, being among the leading causes of death worldwide. In 2021, the disease claimed the lives of 1.6 million people, making it the second leading infectious killer after COVID-19.[1] Drug-resistant TB and long treatment regimens have increased the urgency...
NEW YORK, NY — Parent Project Muscular Dystrophy (PPMD) and the World Duchenne Organization (WDO), along with WDO’s members and affiliates, proudly herald the United Nations marking a momentous milestone for the global rare disease community by officially designating September 7th as World Duchenne Awareness Day (WDAD), set to be...